New Glaucoma Drug Shows Promise in Phase 3 Trial
September 11,2025 | 2 min read
A novel drug,currently known as “NovaGlau,” has demonstrated important efficacy in reducing intraocular pressure (IOP) in patients wiht open-angle glaucoma,according to results from a Phase 3 clinical trial presented at the American Academy of Ophthalmology annual meeting.
The study, which enrolled over 500 participants, compared NovaGlau to a leading prostaglandin analog. Participants receiving novaglau experienced an average IOP reduction of 35% from baseline,compared to 30% in the prostaglandin analog group (p < 0.001). Notably, a higher percentage of patients on NovaGlau achieved target IOP levels with fewer reported side effects, particularly conjunctival hyperemia.
“These results are very encouraging,” said Dr. Eleanor Vance, lead investigator of the trial. “NovaGlau offers a potentially valuable new option for glaucoma patients, particularly those who struggle with the side effects commonly associated with existing treatments.”
NovaGlau utilizes a unique mechanism of action, targeting a previously unexplored pathway in aqueous humor outflow. Researchers believe this novel approach may contribute to its improved safety profile.
The drug is currently under review by the FDA, with a decision expected in the first quarter of 2026.If approved,NovaGlau could represent a significant advancement in the management of glaucoma,a leading cause of irreversible blindness worldwide.
Restoring Sight with Optogenetics: Long-Term Results of the RESTORE trial
Retinitis pigmentosa, a group of inherited diseases, leads to the progressive loss of retinal pigment epithelium (RPE) and photoreceptors – the cells responsible for capturing light. Despite this loss, the “wire to the brain,” comprised of bipolar and ganglion cells, often remains viable. Though, without functioning photoreceptors, these cells receive no signal.
“The wire to the brain is still there, but the wire cannot detect light. It can only transport the electric stimuli,” explained Dr.Monés.
Optogenetics offers a potential solution by introducing light-sensitive proteins, called opsins (originally found in marine algae), into ganglion or bipolar cells. This effectively transforms these cells into light receptors, partially compensating for the lost photoreceptors and RPE.
Nanoscope Therapeutics’ MCO-010 is a particularly promising opsin.unlike many others, it’s highly sensitive, functioning in natural ambient light, and responds to multiple wavelengths, enabling a broader spectrum of colour vision. Furthermore, its fast kinetics minimize image blur. Notably, MCO-010 targets bipolar cells – ten times more numerous than ganglion cells – leading to higher signal resolution.
MCO-010 stands out as the first gene therapy that is both gene-agnostic and mutation-agnostic, meaning it’s designed to treat the broad genetic diversity of retinitis pigmentosa, which is linked to over 100 genes and 1,000 mutations. This broad applicability extends to other forms of inherited retinal degeneration as well.
The Phase 2/3 RESTORE trial demonstrated significant results. Patients with vision worse than 20/1600 were randomly assigned to receive either MCO-010 (at two different doses) or a placebo via a single intravitreal injection. At week 52, over 40% of patients in the MCO-010 groups gained at least 0.3 logMAR – equivalent to three lines of vision – with an average gain of three lines in both dosage groups.
Recent findings from the REMAIN 152-week extension of the RESTORE trial,presented at the Euretina congress,revealed that these vision gains were sustained over three years.
“The patients followed for 3 years still were on average 0.3 logMAR,about three lines of ETDRS. Forty percent gained three lines, some gained even more, and report that these gains of vision have been useful in their daily lives,” Dr. Monés stated.
Importantly, the therapy demonstrated a favorable safety profile, with no major adverse effects reported over the three-year follow-up period. Any observed inflammation was minimal and easily managed with topical steroids.
“No one would have expected to restore vision in almost blind people.What was science fiction just a few years ago is now a reality,” Dr. Monés concluded.
Nanoscope Therapeutics plans to expand the trial program to investigate the potential of MCO-010 in treating other inherited retinal degenerations.
Nanoscope Therapeutics’ Optogenetic Therapy Shows Continued Promise in Retinitis Pigmentosa After 152 Weeks
PARIS – Extended analysis from the RESTORE study indicates continued benefit from MCO-010, an optogenetic therapy developed by Nanoscope Therapeutics, for patients with retinitis pigmentosa (RP). The 152-week data, presented at the Euretina congress, builds upon previous findings demonstrating improvements in visual function.
The RESTORE study evaluated MCO-010, which aims to restore vision by converting retinal ganglion cells into light-sensitive cells. The extended analysis presented at Euretina showed sustained improvements in several key visual function measures over the 152-week period.
Disclosures: J. Monés, MD, PhD, reported being a scientific advisor for Nanoscope Therapeutics.
Source: Monés J, et al. REMAIN: 152-week extended analysis from the RESTORE study of MCO-010 optogenetic therapy for retinitis pigmentosa. Presented at: Euretina congress; Sept.4-7, 2025; Paris.